Mulpleta (lusutrombopag) / Shionogi - LARVOL DELTA

Lusutrombopag improves platelet engraftment after autologous haematopoietic stem cell transplantation in multiple myeloma: A single-centre retrospective study (ASH 2025) - "Result Lusutrombopag accelerated platelet engraftment: Median engraftment time: 10 days (lusutrombopag) vs 12 days (controls) Cumulative incidence at day +13: Significantly higher in lusutrombopag group (P*=0.002; 95% CI: -3.5 to -1.0 days) ≤10-day engraftment rate: Significantly increased (OR=12.5; P*=0.006) Both groups exhibited favourable outcomes with no adverse events. Conclusion Lusutrombopag accelerates platelet engraftment post-ASCT in MM patients, reducing median time by 2 days and transfusion requirements, thereby optimising this critical treatment phase."

Retrospective data • Hematological Malignancies • Multiple Myeloma • Transplantation

FLT3 ligand facilitates long-term ex vivo expansion of human hematopoietic stem cells by maintaining lymphoid reconstitution potential (ASH 2025) - "In a short-term (7-day) culture, we foundthat supplementing SCF in addition to 740 Y-P, or substituting butyzamide with lusutrombopag, an FDA-approved TPO-RA, enhanced the ex vivo expansion of phenotypic hematopoietic stem and progenitorcells (lineage⁻CD41⁻CD34⁺) from CD34+ umbilical cord blood (UCB) cells...In contrast, the 5LS culture demonstrated greater fold expansion of functional HSCs after 14 days(60.6-fold expansion) compared to the 5LSF culture (23.1-fold expansion), which is consistent with theresults from the phenotypic characterization. These findings provide evidence that human functional HSCcan stably expands long-term in the 5LSF culture.In conclusion, we have developed a long-term expansion protocol for human HSCs that facilitates bothclinical and research applications of these functional stem cells."

Preclinical • Hematological Disorders • Inflammation • CD33 • CD34 • FLT3 • ITGA2B • ITGA3 • PROCR • STAT5 • THY1

Prolonged thrombocytopenia following diverse CAR-T cell therapies: High incidence, inflammatory risk factors, and a comparative efficacy analysis of TPO-receptor agonist strategies in a real-world cohort (ASH 2025) - "The efficacy of TPO-RA interventions—includingmonotherapy (Eltrombopag, Avatrombopag, Hetrombopag, Lusutrombopag, Romiplostim, or rhTPO) andcombination strategies—was evaluated. Prolonged, severe thrombocytopenia is a frequent, inflammation-driven complication of CAR-T therapy. Our real-world data demonstrate a clear efficacy hierarchy among TPO-RAs, with romiplostimbeing the most effective single agent. Crucially, combination TPO-RA strategies, particularly thoseinvolving romiplostim or sequential rhTPO, significantly accelerate platelet recovery in severe caseswithout increasing toxicity."

CAR T-Cell Therapy • Clinical • Real-world • Real-world evidence • B Cell Non-Hodgkin Lymphoma • Cardiovascular • Hematological Disorders • Hematological Malignancies • Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Thrombocytopenia • IFNG

Efficacy and safety of lusutrombopag monotherapy for cyclosporine A-refractory, transfusion-dependent non-severe aplastic anemia (PubMed, Zhonghua Xue Ye Xue Za Zhi) - "During follow-up, one patient experienced a loss of OR after discontinuing treatment, with a relapse rate of 14.3%; no clonal evolution or mortality was observed. These findings suggest that lusutrombopag is both effective and well-tolerated in CsA-refractory TD-NSAA patients and represents a promising therapeutic option for those with poor treatment tolerability."

Journal • Monotherapy • Retrospective data • Anemia • Aplastic Anemia • Hematological Disorders

Lusutrombopag or hetrombopag supports in vitro megakaryopoiesis better than other thrombopoietin receptor agonists. (PubMed, Br J Haematol) - No abstract available

Journal • Preclinical

A Prospective Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of Lusutrombopag in Chinese Adults with Persistent or Chronic Primary Immune Thrombocytopenia (ASH 2024) - P2 | "TPO-RAs (romiplostim, eltrombopag, hetrombopag, and avatrombopag) has been approved for treating ITP in Chinese...Prior to enrollment, 50% had been receiving stable steroids (same milligram amount ±10% and ≤ 20mg of equivalent dose of prednisone) for ≥2 weeks and no other concomitant medications affecting PLTs were administered...Treatment emergent adverse events (TEAEs) occurred in four of eight patients and there were no treatment-related adverse reactions. None experienced serious adverse events (SAE).Conclusions : Lusutrombopag, a newly approved TPO-RA in China, has shown preliminary efficacy initiated with a once-daily 3mg tablet with tolerability in Chinese adults with persistent or chronic primary ITP."

Clinical • Hematological Disorders • Hepatology • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura

Development and validation of a prognostic prediction model based on coagulation-related genes and clinical factors in acute leukemia. (PubMed, Transl Pediatr) - "The model helps to enable individualised risk stratification and guide targeted therapy. In addition, avatrombopag and lusutrombopag as potential therapeutic agents provide new ideas for precision medicine in paediatric AL."

Journal • Hematological Malignancies • Leukemia • Oncology • Pediatrics • PROS1

Relationship between thromboembolic events and thrombopoietin receptor agonists: a pharmacovigilance analysis of the FDA Adverse Event Reporting System and the Japanese Adverse Drug Event Report. (PubMed, BMJ Open) - "The study provided real-world evidence of TEE associated with TPO-RAs, highlighting a strong link despite variations in signal values and regional reporting practices. Findings underscore ongoing clinical safety surveillance for TPO-RAs."

Adverse events • Journal • Retrospective data • Cardiovascular • Hematological Disorders • Thrombocytopenia

An open-lable, single-arm pilot study to evaluate the efficacy and safety of lusutrombopag in Chinese adults with persistent or chronic immune thrombocytopenia (ChiCTR) - P4 | N=17 | Completed | Sponsor: Institute of Hematology& Blood Disease Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College; Institute of Hematology&a | Recruiting ➔ Completed

Trial completion • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Efficacy and safety of lusutrombopag in the treatment of patients with non-severe aplastic anemia (NSAA). (ChiCTR) - P4 | N=15 | Recruiting | Sponsor: Tianjin Medical University General Hospital; Tianjin Medical University General Hospital | Not yet recruiting ➔ Recruiting

Enrollment open • Anemia • Aplastic Anemia • Hematological Disorders

Pharmacist intervention through protocol-based pharmacotherapy management is effective to ensure safety in invasive procedures for chronic liver disease. (PubMed, J Pharm Health Care Sci) - "The use of platelet products decreases without the increased incidences of hemorrhage if pharmacists suggest prescribing thrombopoietin receptor agonists based on their assessment of the platelet count and the bleeding risk of the procedure."

Journal • Hematological Disorders • Hepatology • Thrombocytopenia

Lusutrombopag combined with recombinant human thrombopoietin for the treatment of thrombocytopenia in patients with chronic liver disease destined to undergo elective invasive surgery: a prospective, multicentre, single-arm clinical study (ChiCTR) - P2 | N=60 | Not yet recruiting | Sponsor: THE FIRST AFFILIATED HOSPITAL OF USTC (ANHUI PROVINCAL HOSPITAL); THE FIRST AFFILIATED HOSPITAL OF USTCANHUI PROVINCAL HOSPITAL

New P2 trial • Hematological Disorders • Hepatology • Thrombocytopenia

Usefulness of Repeated Lusutrombopag Administration in Clinical Practice and its Correlation with the Simulation Software-Predicted Platelet Count: A Multicenter Study. (PubMed, Intern Med) - "There was a correlation between the highest software-predicted platelet count and the platelet count measured in actual clinical practice in all dose frequency groups; however, the correlation was the strongest in the one-dose group (r = 0.74, p <0.0001). Conclusion Frequent lusutrombopag administration is therefore both effective and safe, and a platelet prediction simulation software program is thus considered to be useful in practice."

Clinical • Journal • Hematological Disorders • Hepatology • Immunology • Liver Cancer • Oncology • Solid Tumor • Thrombocytopenia

QSAR-Based Drug Repurposing and RNA-Seq Metabolic Networks Highlight Treatment Opportunities for Hepatocellular Carcinoma Through Pyrimidine Starvation. (PubMed, Cancers (Basel)) - "For DHODH, the drugs Oteseconazole, Tipranavir, and Lusutrombopag were identified as potential inhibitors. For TYMS, the drugs Tadalafil, Dabigatran, Baloxavir Marboxil, and Candesartan Cilexetil showed promise as inhibitors. Overall, this study suggests in vitro testing of the identified drugs to assess their capabilities in inducing pyrimidine starvation on HCC."

Journal • Hepatocellular Cancer • Oncology • Solid Tumor • TYMS

Clinical research progress of lusutrombopag for the treatment of thrombocytopenia in chronic liver disease (PubMed, Zhonghua Gan Zang Bing Za Zhi) - "Lusutrombopag is a newly developed small-molecule TPO receptor agonist that can induce bone marrow progenitor cells to differentiate into megakaryocytes and increase platelet production, posing characteristics of oral convenience, safety and effectiveness; thus, it has been used in many countries and regions for the treatment of patients with chronic liver diseases concurrent with thrombocytopenia. Herein, the pharmacological features and clinical research are reviewed."

Journal • Review • Hematological Disorders • Hepatology • Thrombocytopenia

Lusutrombopag Combined With Recombinant Human Thrombopoietin for the Treatment of Thrombocytopenia in Patients With Chronic Liver Disease Destined to Undergo Elective Invasive Surgery (clinicaltrials.gov) - P2 | N=60 | Recruiting | Sponsor: Anhui Provincial Hospital

New P2 trial • Surgery • Hematological Disorders • Hepatology • Thrombocytopenia

An open-lable, single-arm pilot study to evaluate the efficacy and safety of lusutrombopag in Chinese adults with persistent or chronic immune thrombocytopenia (ChiCTR) - P4 | N=17 | Recruiting | Sponsor: Institute of Hematology& Blood Disease Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College; Institute of Hematology&a | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Asymmetric Transfer Hydrogenation of Ketones Improved by PNN-Manganese Complexes. (PubMed, J Org Chem) - "Notably, Mn2 proved to be the most productive catalyst, allowing an outstanding turnover number of 8300 with catalyst loadings as low as 0.01 mol %. Furthermore, this catalytic protocol shows considerable promise for applications in the synthesis of chiral drugs such as Lusutrombopag."

Journal

Lusutrombopag in the Treatment of Immune Thrombocytopenia (ITP) (clinicaltrials.gov) - P2 | N=17 | Recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital, China | Phase classification: P4 ➔ P2

Phase classification • Hematological Disorders • Thrombocytopenia • Thrombocytopenic Purpura

Final Results from the First European Real-World Experience on Lusutrombopag Treatment in Cirrhotic Patients with Severe Thrombocytopenia: Insights from the REAl-World Lusutrombopag Treatment in ITalY Study. (PubMed, J Clin Med) - " In this first European real-world series, lusutrombopag demonstrated efficacy and safety consistent with the results of registrational studies. According to our results, patients with baseline platelet counts ≤29,000/μL are unlikely to respond to the drug."

Journal • Real-world • Real-world evidence • Cardiovascular • Hematological Disorders • Hepatology • Liver Cirrhosis • Thrombocytopenia • Thrombosis • Venous Thromboembolism

Lusutrombopag in the Treatment of Immune Thrombocytopenia (ITP) (clinicaltrials.gov) - P4 | N=17 | Recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital, China | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Disorders • Thrombocytopenia • Thrombocytopenic Purpura

Update on the Use of Thrombopoietin-Receptor Agonists in Pediatrics. (PubMed, Hamostaseologie) - "Romiplostim and eltrombopag are already established as second-line treatment for persistent or chronic immune thrombocytopenia (ITP). Current developments include testing of avatrombopag and lusutrombopag that are approved for the treatment of thrombocytopenia associated with chronic liver disease (CLD) in adult patients. In pediatric and adolescent medicine, we expect in the near future a broader use of TPO-RAs as first-line treatment in primary ITP, thereby considering immunomodulatory effects that increase the rate of sustained remission off-treatment, and a selective use in rare inherited thrombocytopenias based on current clinical trials."

Journal • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Hepatology • Immunology • Myelodysplastic Syndrome • Oncology • Pediatrics • Primary Immunodeficiency • Thrombocytopenia • Thrombocytopenic Purpura • Transplantation

EFFICACY AND SAFETY OF HETROMBOPAG IN INCREASING PLATELET COUNTS IN PATIENTS WITH HEPATOCELLULAR CARCINOMA UNDERGOING HEPATECTOMY: A RANDOMIZED, PLACEBO-CONTROLLED STUDY (EHA 2024) - "Similar agents like avatrombopag and lusutrombopag havebeen approved for TCP in chronic liver disease patients undergoing a procedure. Hetrombopag reduced the need for platelet transfusion or any other intervention for TCP compared toplacebo, with acceptable safety. HPAG may offer an effective and safe alternative to platelet transfusions inHCC patients who require increased PC for scheduled hepatectomy."

Clinical • Anemia • Aplastic Anemia • Cardiovascular • Gastrointestinal Cancer • Hematological Disorders • Hepatocellular Cancer • Hepatology • Oncology • Solid Tumor • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis • Venous Thromboembolism

Final results from the italian real-world experience on lusutrombopag treatment in cirrhotic patients with severe thrombocytopenia: insights from the reality study (EASL-ILC 2024) - "In this first real-world European series of CLD, lusutrombopag demonstrated efficacy and safety consistent with findings from registrative trials. Finally, according to our results, patients with baseline platelets ≤29.000/μL are unlikely to respond to the drug."

Clinical • Real-world • Real-world evidence • Cardiovascular • Hematological Disorders • Infectious Disease • Inflammation • Liver Cirrhosis • Thrombocytopenia • Thrombosis • Venous Thromboembolism

Prevention of bleeding related to invasive procedures in patients with cirrhosis (EASL-ILC 2024) - "Large trials examining avatrombopag18 and lusutrombopag19 enrolled patients with cirrhosis and thrombocytopenia undergoing procedures and randomised them to receive TPO-R agonist vs. placebo. Future collaborative multicentre clinical research will bridge remaining knowledge gaps. In the meanwhile, hepatologists should work to promote guideline recommendations broadly among clinicians to improve and standardise care of patients with cirrhosis."

Clinical • Cardiovascular • Fibrosis • Hematological Disorders • Hepatology • Immunology • Infectious Disease • Inflammation • Liver Failure • Portal Hypertension • Thrombocytopenia • Thrombosis